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Recruiting Phase 3

Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

NCT05935215 · tracked via the Priya Life Science Italy tracker
Phase
Phase 3
Started
2024-02-28
Last updated
2026-04-29

Condition(s) studied

Atypical Hemolytic Uremic Syndrome

Investigational drug(s) / intervention(s)

Iptacopan →

Iptacopan: Open Label

Study summary

The purpose of this Phase 3 study is to evaluate the efficacy and safety of iptacopan upon switching from anti-C5 antibody to iptacopan treatment in study participants with aHUS.

Eligibility

Sex
ALL
Min age
18 Years
Max age
100 Years
Healthy volunteers
No
Inclusion Criteria: * Male and female adult participants ≥ 18 years of age with diagnosis of aHUS for whom etiologies of other types of TMA and non-aHUS kidney disease have been excluded. •. Currently on the recommended (as per label) dosage regimen of anti-C5 antibody treatment, for at least 3 months prior to entering the screening period. * In the opinion of the investigator the participant has responded to anti-C5 antibodytreatment prior to screening and has clinical evidence of response (in absence of PE/PI) during the Screening period. Clinical evidence of response to anti-C5 antibody treatment (in absence of PE/PI) confirmed during the Screening period by central laboratory at two visits 12 weeks apart. Clinical evidence of response is defined as: 1. Hematological normalization in platelet count ≥150 x 10\^9/L and LDH below upper limit of normal \[ULN\], and 2. Stable kidney function as defined by serum creatinine values within ±15% during the Screening period * Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infections is required prior to the start of treatment with iptacopan. * If not received previously or if a booster is required, vaccination against Haemophilus influenzae infection, should be given, if available and according to local regulations. Exclusion Criteria: * History of aHUS disease relapse while on anti-C5 antibody treatment. * eGFR \< 30 ml/min/1.73m\^2 * Active infection or history of recurrent invasive infections caused by encapsulated bacteria, i.e., meningococcus, pneumococcus (eg., N. meningitidis, S. pneumoniae) or H. influenzae. * Participants with sepsis or active systemic bacterial, viral (including COVID-19) or fungal infection within 14 days prior to study treatment administration. * Kidney, bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT), heart, lung, small bowel, pancreas, liver transplantation or any other cell or solid organ transplantation * Female patients who are pregnant or breastfeeding, or intending to conceive during the course of the study * Any medical condition deemed likely to interfere with the patient's participation in the study

Primary outcome measure(s)

  • Percentage of participants free of TMA manifestation — 12 months
    Absence of thrombotic microangiopathy (TMA) manifestation, without use of anti-C5 antibody, during the 12 months of iptacopan treatment following the switch of treatment from an anti-C5 antibody to iptacopan treatment.

Trial sites (31)

FacilityCityRegionStatus
Novartis Investigative Site Nanjing Jiangsu Withdrawn
Novartis Investigative Site Beijing China Active Not Recruiting
Novartis Investigative Site Beijing China Active Not Recruiting
Novartis Investigative Site Shanghai China Withdrawn
Novartis Investigative Site Bordeaux France Recruiting
Novartis Investigative Site Paris France Recruiting
Novartis Investigative Site Paris France Recruiting
Novartis Investigative Site Rouen France Recruiting
Novartis Investigative Site Toulouse France Recruiting
Novartis Investigative Site Tours France Recruiting
Novartis Investigative Site Essen Germany Recruiting
Novartis Investigative Site Kiel Germany Recruiting
Novartis Investigative Site Ranica BG Recruiting
Novartis Investigative Site Milan MI Recruiting
Novartis Investigative Site Roma RM Recruiting
Novartis Investigative Site Matsumoto-shi Nagano Recruiting
Novartis Investigative Site Iruma-gun Saitama Recruiting
Novartis Investigative Site Bunkyo Ku Tokyo Recruiting
Novartis Investigative Site Santiago Compostela A Coruna Recruiting
Novartis Investigative Site Barcelona Spain Recruiting
Novartis Investigative Site Córdoba Spain Recruiting
Novartis Investigative Site Málaga Spain Recruiting
Novartis Investigative Site Seville Spain Recruiting
Novartis Investigative Site Valencia Spain Recruiting
Novartis Investigative Site Izmir Balcova Withdrawn
Novartis Investigative Site Köseköy Kocaeli Recruiting
Novartis Investigative Site Ankara Yenimahalle Recruiting
Novartis Investigative Site Mersin Yenisehir Recruiting
Novartis Investigative Site Glasgow Scotland Recruiting
Novartis Investigative Site Newcastle upon Tyne Tyne and Wear Recruiting
Novartis Investigative Site London United Kingdom Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05935215 on ClinicalTrials.gov ↗ ← All trials in Italy